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Article

GLP-1R agonists demonstrate potential to treat Wolfram syndrome in human preclinical models

30 Mar 2023

Abstract excerpt

AIMS/HYPOTHESIS: Wolfram syndrome is a rare autosomal recessive disorder caused by pathogenic variants in the WFS1 gene. It is characterised by insulin-dependent diabetes mellitus, optic nerve atrophy, diabetes insipidus, hearing loss and neurodegeneration. Considering the unmet treatment need for this orphan disease, this study aimed to evaluate the therapeutic potential of glucagon-like peptide 1 receptor...

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PMID: 36995380
DOI: 10.1007/s00125-023-05905-8

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